Mitochondrial disease, where the tiny energy factories in cells aren’t functioning properly, can have devastating and sometimes fatal consequences. As there are no approved treatments, patients rely on strategies such as strict diets and conserving energy to try and control symptoms that can affect organs including the brain, kidneys, muscles, pancreas and heart. Netherlands-based biotech Khondrion BV is one of a group of companies that is making progress against this group of rare inherited conditions that affects around one in every 4,000 people.
Biogen Inc. opened the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) virtual meeting with data showing those treated with its multiple sclerosis (MS) therapies had an effective antibody response to COVID-19 vaccination.
DUBLIN – Shares in Idorsia Ltd. were off almost 4% Oct. 11 on news that lucerastat missed the primary endpoint of the Modify phase III trial in Fabry disease. The double-blinded, placebo-controlled study, which recruited 118 adult patients, was designed to evaluate the effect of lucerastat on neuropathic pain. Patients were randomized to drug or placebo in a 2-to-1 ratio.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: 4D, Aligos, Astrazeneca, Bicycle, Diurnal, Idorsia, Galmed, Oxurion, Regenxbio, Reistone, Repertoire, Sarepta.
On the last day of this year’s Molecular Targets meeting, an annual joint conference of the American Association for Cancer Research, the National Cancer Institute and the European Organization for the Research and Treatment of Cancer, the final plenary went from molecular to macro in a lively discussion of the biggest roadblock in cancer drug development, and what can be done to improve it.
Shares of Protagonist Therapeutics Inc. (NASDAQ:PTGX) climbed 93.9% on Oct. 11 after the FDA removed a full clinical hold on studies of the rusfertide, the company's investigational treatment for the blood disorders polycythemia vera and hereditary hemochromatosis. Triggered by a finding of malignant skin tumors in mice treated with the drug disclosed on Sept. 17, the FDA's three-weeks-ago hold had pushed Protagonist shares down by as much as 72%.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Acumen, Argenx, Cyprium, Fortress, Nanoscope, Nkarta.
Allogene Therapeutics Inc. officials took many questions but had few answers during a conference call regarding the FDA’s clinical hold after the report of a chromosomal abnormality in ALLO-501A CAR T cells in a patient treated in the phase I/II Alpha2 study.
It looks like Shanghai Miracogen Inc.’s antibody-drug conjugate (ADC) partnership with Synaffix BV is paying off as MRG-004A, an ADC designed to treat solid tumors, has entered a U.S. phase I/II trial to treat solid tumors.
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